Clinical trials: Bone Marrow Failure, active
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Severe Aplastic Anemia, Hypo-Plastic MDS
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.
- Conditions
- Severe Aplastic Anemia, Hypo-Plastic MDS, Myelodysplastic Syndrome (MDS)
- Trial name
- NCT03173937
- Testing
- Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent System
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Acute Leukemia, Myelodysplastic Syndromes
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled.
- Conditions
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia (SAA)
- Trial name
- NCT07796620
- Testing
- Sirolimus, Ruxolitinib, MMF
- Volunteers
- 40 people
- Sponsor
- Peking University People's Hospital
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia.
- Trial name
- NCT01174108
- Testing
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia, Aplastic Anaemia
A Novel Conditioning Regimen for Haplo-HSCT in Older Patients With SAA
The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.
- Conditions
- Severe Aplastic Anemia, Aplastic Anaemia, Hematopoietic Cell Transplant
- Trial name
- NCT07586735
- Testing
- BFCA conditiong regimen
- Where
- China
- Volunteers
- 64 people
- Sponsor
- Peking University People's Hospital
Severe Aplastic Anemia
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding.
- Trial name
- NCT02828592
- Testing
- Fludarabine, Cyclophosphamide, Total Body Irradiation
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Northside Hospital, Inc.
Aplastic Anemia, Refractory
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
This study consists of two phases, phase Ib and phase II. The primary objective of phase Ib is to evaluate the safety and tolerability of multiple doses of daratumumab in patients with refractory aplastic anemia.
- Conditions
- Aplastic Anemia, Refractory, Relapse
- Trial name
- DARA-AA-001 (NCT07287228)
- Testing
- Daratumumab
- Where
- China
- Volunteers
- 37 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hematologic Malignancy, Bone Marrow Transplant
Tocilizumab for the Prevention of Graft Failure and GVHD in Haplo-Cord Transplantation
The purpose of this study is to evaluate the safety of reducing and ultimately eliminating anti-thymocyte globulin (ATG) from the haplo-cord transplant conditioning regimen and replacing it with tocilizumab, an IL-6 receptor…
- Trial name
- NCT04395222
- Testing
- Tocilizumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Weill Medical College of Cornell University
Aplastic Anemia
Revolade Tablets Specified Drug-use Survey
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).
- Trial name
- NCT06287268
- Testing
- eltrombopag
- Where
- 17 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia, Cytopenia
A Study of Emapalumab for Pediatric Aplastic Anemia
The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD
- Conditions
- Aplastic Anemia, Cytopenia, Hypocellular Marrow
- Trial name
- NCT06430788
- Testing
- Emapalumab
- Where
- 6 sites in United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Acute Myeloid Leukemia in Remission, Adult Acute Lymphoblastic Leukemia in Complete Remission
Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant
This phase Ib/2 trial studies how well chemotherapy, total body irradiation, and post-transplant cyclophosphamide work in reducing rates of graft versus host disease in patients with hematologic malignancies undergoing a donor…
- Conditions
- Acute Myeloid Leukemia in Remission, Adult Acute Lymphoblastic Leukemia in Complete Remission, Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission, Chronic Myelomonocytic Leukemia in Remission
- Trial name
- NCT03192397
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Fludarabine Phosphate
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Roswell Park Cancer Institute
Aplastic Anemia
Long Term Follow-up Observational Study After Clinical Trials of AMG531 (Romiplostim) in Patients With Untreated Aplastic Anemia
To evaluate the long-term efficacy and safety after the end of romiplostim treatment by observation [up to 5 years] of patients who were registered for Study 531-003/531-004 in immunosuppressive therapy-naïve patients with…
- Trial name
- NCT04870346
- Where
- Japan
- Volunteers
- 36 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Dyskeratosis Congenita, Telomere Disease
Quercetin Dyskeratosis Congenita (DC)/Telomere Biology Disorders (TBD)
The purpose of this study is to see if a vitamin-like substance called quercetin is safe for people who have a rare condition called Dyskeratosis congenita (DC) or telomere biology disorders (TBD).
- Trial name
- NCT07628972
- Testing
- Quercetin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Acute Leukemia, Immune Deficiency Disorder
Umbilical Cord Blood Transplantation From Unrelated Donors
This study is being done to determine how long it takes for the engraftment (recovery of blood cell counts) of umbilical cord stem cells and also how often engraftment of umbilical cord stem cells transplanted from an unrelated…
- Conditions
- Acute Leukemia, Immune Deficiency Disorder, Congenital Hematological Disorder, Metabolism Disorder
- Trial name
- NCT03016806
- Testing
- Total Body Irradiation 1200 cGy, Total Body Irradiation 200 cGy, Cyclophosphamide
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Rochester
Acute Leukemia, Acute Myeloid Leukemia
Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders
This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine/busulfan for patients unable to receive further…
- Conditions
- Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Lymphoma
- Trial name
- NCT03314974
- Testing
- HSCT with TBI Regimen, HSCT with Non-TBI Regimen
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio